Agalsidase alfa in pediatric patients with Fabry disease: a 6.5-year open-label follow-up study.

ABSTRACT:

Background

Signs and symptoms of the X-linked disorder, Fabry disease (FD), can occur early during childhood with heterogeneous clinical manifestations including potential cardiac and renal dysfunction. Several studies support the efficacy of the enzyme replacement therapy (ERT) agalsidase alfa, in adults with FD, though published data on the long-term safety and efficacy of agalsidase alfa in children are limited. As early treatment with ERT has the potential to reduce complications arising from disease progression, children in particular could benefit. The objective of this study was to evaluate the safety and efficacy of long-term agalsidase alfa ERT in children with FD.

Methods

TKT029 was a 6.5-year open-label, multicenter, extension study of children who completed TKT02

SUBMITTER: Schiffmann R 

PROVIDER: S-EPMC4260255 | biostudies-literature | 2014 Nov

REPOSITORIES: biostudies-literature